Trial of the Combined Use of Thiamine and Biotin in Patients With Huntington's Disease

NCT04478734 | Recruiting Phase 2

• 1 other identifier: HUNTIAM
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 3

Brief Summary

Evaluate the safety and tolerability of combined oral thiamine with biotin therapy in patients with Huntington´s disease in mild to moderate stages and it is intended to evaluate the biological effect of the treatment in the central nervous system of these patients using as the main biomarker the increase in the level of thiamine monophosphate (TMP) in cerebrospinal fluid (CSF) of these patients with Huntington Disease (HD) during a follow-up period of one year. Our main hypothesis is that combined thiamine-biotin oral therapy is a secure and well-tolerated treatment, potentially capable of modifying the disease course or avoiding the progression of symptoms in early-stages HD patients

Conditions

Huntington Disease

Keywords

thiamine and biotine; combination therapy

Outcome Measures

Primary Outcomes (1)

• Incidence of Treatment-Emergent Adverse Events as assessed by clinical examination anamnesis and Analytical monitoring with hematological and biochemical control (hepatic and renal function)

  Patient´s condition and emergence of comorbidity by clinical examination and anamnesis directed by a neurologist, by measuring of vital signs (blood pressure, heart rate,breath rate weight and height)

  From signature of informed consent form, at all scheduled visits, to end of follow up week 52

Secondary Outcomes (1)

• The evaluation of the efficacy of treatment with combined oral thiamine and biotin therapy in increasing thiamine monophosphate (TMP) levels in CSF of patients with HD

  At baseline (week 0) and visit 8 (week 48)

Other Outcomes (6)

• Evaluate the biological effect of the combined thiamine-biotin oral therapy in the neurodegeration of HD patients

  At baseline (week 0) and visit 8 (week 48)

• Evaluate the biological effect of the combined thiamine-biotin oral therapy in the neuroimaging progression markers in patients with HD

  At baseline (week 0) and visit 8 (week 48)

• Evaluate the effect of the combined thiamine-biotin oral therapy in the quality of life of patients with HD

  At baseline (week 0), week 24 and week 48

Study Arms (2)

Moderate doses

EXPERIMENTAL

moderate doses of combination therapy applying the minimum average dosage of thiamine and biotin used in patients with BTBGD

Drug: Moderate doses of Thiamine y Biotin

High doses

EXPERIMENTAL

high doses of the combination therapy applying the average standard dosage of thiamine and biotin used in patients with BTBGD.

Drug: High doses of Thiamine y Biotin

Interventions

Moderate doses of Thiamine y Biotin DRUG

Thiamine 600 mg every day + Biotin 150mg every day

Also known as: moderate doses of combination therapy

Moderate doses

High doses of Thiamine y Biotin DRUG

Thiamine 1200 mg every day + Biotin 300mg every day

Also known as: high doses of the combination therapy

High doses

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients of legal age with manifest Huntington's disease with motor symptoms (chorea, dystonia or bradykinesia) and/or neuropsychiatric; and genetic confirmation of a number of repetitions of the cytosine-adenine-guanine trinucleotide (CAG triplet) in the HTT gene (coding for HTT) greater than or equal to 39
• Patients should be capable of giving informed consent and attending the planned visit of the study.
• Women of childbearing age should obtain a negative result in the serum or urine pregnancy test at the screening visit. They must also accept the use of appropriate contraceptive methods during the course of the clinical trial and men who have a partner of childbearing age, accept the use of contraceptive methods

You may not qualify if:

• Medical comorbidities considered clinically significant by the clinical judgment of the investigators.
• Pregnancy or lactation
• Patients with HD dependents on the basic routine daily life activities (UHDRS TFC \< 7) or a severe cognitive decline.
• Active psychosis at the moment of the screening evaluation.
• Severe renal failure.
• Patients previously treated with thiamine and/or biotin or enrolled in other HD clinical trial with oligonucleotide antisense (IONIS-HTTRX (RG6042).

Sponsors & Collaborators

• Fundación Pública Andaluza para la gestión de la Investigación en Sevilla: lead
• Ciberned (Centro de Investigación Biomédica en Red): collaborator

Study Sites (3)

Hospital Universitario de San Sebastián

San Sebastián, San Sebastian, 20014, Spain

RECRUITING

Virgen del Rocío Hospital

Seville, Seville, 41013, Spain

RECRUITING

Hospital Ramón y Cajal

Madrid, Spain

RECRUITING

MeSH Terms

Conditions

Huntington Disease

Condition Hierarchy (Ancestors)

Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Dementia; Chorea; Dyskinesias; Movement Disorders; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Cognition Disorders; Neurocognitive Disorders; Mental Disorders

Study Officials

• Pablo Mir Rivera, MD/PhD

  Institute of Biomedicine of Seville (IBiS)

  STUDY DIRECTOR

• Clara M. Rosso Fernández

  Virgen del Rocío University Hospital Research and Clinical Trials Unit

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Pablo Mir Rivera, MD/PhD

CONTACT

+34 955923039; pmir@us.es

Clara M. Rosso Fernández, MD/PhD

CONTACT

+34 955012144; claram.rosso.sspa@juntadeandalucia.es

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 11, 2020
• First Posted: July 21, 2020
• Study Start: April 12, 2023
• Primary Completion: June 30, 2025
• Study Completion: December 30, 2025
• Last Updated: February 25, 2025

Record last verified: 2025-01

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, CSR
• Time Frame: After the primary completion date and submit results information
• Access Criteria: collaborating researchers

Locations

ES (3)